A Phase 1 Study to Evaluate the Pharmacokinetics and Tolerability of Oral Azacitidine in Japanese Patients With Myelodysplastic Syndromes

Phase 1

Completed

• Conditions: Myelodysplastic Syndromes
• Interventions: Drug: Oral azacitidine

• Registration Number: NCT01571648
• Lead Sponsor: Celgene

Brief Summary

The purpose of this study is to evaluate the tolerability of oral azacitidine in the treatment of patients with Myelodysplastic Syndromes (MDS).

Detailed Description

Not available

Study Timeline

• Study Start: 2012-04-01
• Study First Submitted: 2012-04-03
• Study First Submitted QC: 2012-04-04
• Study First Posted: 2012-04-05
• Primary Completion: 2013-01-01
• Study Completion: 2013-01-01
• Status Verified: 2019-11
• Last Update Submitted: 2019-11-07
• Last Update Posted: 2019-11-12

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 5

Inclusion Criteria

Patients must satisfy the following criteria to be enrolled in the study:

• Have a documented diagnosis of myelodysplastic syndromes (MDS) according to World Health Organization (WHO) 2008 classification
• Age ≥ 20 years;
• Written informed consent;
• Eastern Cooperative Oncology Group (ECOG) performance status of 0-2;
• Resolution of any toxic effects of prior anti-cancer therapy; and
• Negative urine or serum pregnancy test on females of childbearing potential.

Exclusion Criteria

The presence of any of the following will exclude a patient from enrollment:

• Treatment with chemotherapy, radiotherapy, or surgery within 4 weeks of study registration;
• Pregnant or breast-feeding females;
• Previous or concomitant malignancy other than MDS;
• Significant active cardiac disease within the previous 6 months;
• Uncontrolled systemic infection or
• Known infection with human immunodeficiency virus (HIV), hepatitis B, or hepatitis C

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Oral azacitidine	Oral azacitidine	-

Primary Outcome Measures

Name	Time	Method
Incidence of dose-limiting toxicity in accordance with Common Terminology Criteria for Adverse Events	1 month	Incidence of dose-limiting toxicity in accordance with Common Terminology Criteria for Adverse Events

Secondary Outcome Measures

Name	Time	Method
PK- Maximum concentration in plasma (Cmax)	0, 0.25, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 6, 8 hours post-dose	PK- Maximum concentration in plasma (Cmax)
PK-Terminal half-life (T1/2,z)	0, 0.25, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 6, 8 hours post-dose	PK-Terminal half-life (T1/2,z)
PK-Apparent total body clearance (CL/F)	0, 0.25, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 6, 8 hours post-dose	PK-Apparent total body clearance (CL/F)
Safety (type, frequency, severity, number of participants with adverse events)	Up to 2 years	Safety (type, frequency, severity, number of participants with adverse events)
PK-Apparent volume of distribution (Vz/f)	0, 0.25, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 6, 8 hours post-dose	PK-Apparent volume of distribution (Vz/f)
Efficacy (Hematologic response and hematologic improvement)	Up to 2 years	Efficacy (Hematologic response and hematologic improvement)
PK-Area under the plasma concentration-time curve (AUC)	0, 0.25, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 6, 8 hours post-dose	PK-Area under the plasma concentration-time curve (AUC)
PK- Time to maximum plasma concentration (Tmax)	0, 0.25, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 6, 8 hours post-dose	PK- Time to maximum plasma concentration (Tmax)
PK-Elimination rate constant (Kel)	0, 0.25, 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 6, 8 hours post-dose	PK-Elimination rate constant (Kel)

Trial Locations

Locations (1)

Celgene Trial Site; 🇯🇵 Tokyo, Japan